Asmacure Announces Top‐Line Clinical Results for ASM‐024 DPI in Ph 1/2a Trial ‐‐Data indicate ASM‐024 DPI safe and generally well‐tolerated in asthma‐‐
Quebec City, Canada – June 18, 2014 – Asmacure Ltée, a clinical‐stage biopharmaceutical company focused on the development of novel, proprietary compounds that target cholinergic receptors for the treatment of pulmonary diseases, today announced top‐line results from a combined Phase 1 and 2a trial evaluating its lead compound, ASM‐024, in moderate asthma patients.
The data comes from Asmacure’s recently completed Phase 1 single‐ascending dose study in healthy volunteers and a Phase 2a safety, tolerability and pharmacokinetic (PK) study utilizing the dry powder for inhalation (DPI) formulation of ASM‐024 in 15 patients with moderate asthma.
“The data from this trial showed that ASM‐024 at therapeutic doses below 20 mg was generally safe and well tolerated and support further exploration of an effect of the ASM‐024 DPI alone or as an adjunctive treatment with standard of care in patients with moderate and severe asthma,” said Yvon Cormier, M.D., Asmacure’s founder and chief medical officer. “The pharmacokinetics data from this study provided important information on the potential dose range for ASM‐024 in the treatment of the moderate asthma patient when delivered as a dry powder for inhalation.”
ASM‐024 is under development in a dry powder for inhalation (DPI) dosage form. Typically, DPI’s are a preferred dosage form for pulmonary products because they present the potential for delivery of lower, more optimal dosing with greater lung deposition and improved patient tolerability.
“Following this study where ASM‐024 was studied for the first time in the dry powder for inhalation formulation in humans, these trial data support further development of the ASM‐024 DPI in a Phase 2 program with a longer duration of treatment in the moderate and severe asthma population,” said Martin Driscoll, chief executive officer. “We will determine our next steps for the development of the ASM‐024 DPI once we conclude our trial later this year in moderate to severe COPD where we have dosed patients up to two weeks.”
Study Overview Phase 1
- Single‐ascending dose in 40 healthy volunteers; eight subjects received placebo and 32 were dosed with ASM‐024 DPI
- Single‐ascending doses of the ASM‐024 DPI ranged from 1 mg – 60 mg
- Safety, tolerability, and PK study utilizing the dry powder for inhalation (DPI) formulation of ASM‐024 in patients with moderate asthma
- Doses ranged from 1 – 30 mg
- Placebo‐controlled, dose escalation study with the ASM‐024 DPI administered to 15 patients with moderate asthma at a single Investigator Site
- Study design included a total of three visits to the Investigator Clinical Site with each treatment day separated by 7 days (+/‐ one day)
- Each patient received a placebo and up to two ascending doses of the ASM024 DPI on each visit to the site Results
- There were no serious adverse events (SAEs) throughout the duration of the trial
- ASM‐024 DPI was shown to be generally well‐tolerated and safe in the moderate asthma population at doses of < 20 mg
- The most common adverse events were cough and taste
- Systemic exposure was observed in all patients at doses of 3 mg and higher
Asthma is a reversible obstructive lung disease, caused by increased reaction of the airways to various stimuli. It is a chronic inflammatory condition with acute exacerbations. Asthma can be a life‐threatening disease if not properly managed. According to the American Lung Association, in 2011 it was estimated that 25.9 million Americans currently have asthma, including 7.1 million children under 18. Of these, 13.2 million Americans (4.1 million children) had an asthma attack. Close to 2.1 million emergency room visits were attributed to asthma in 2009.
Chronic obstructive pulmonary disease (COPD) refers to a group of lung diseases characterized by chronically poor airflow that typically worsens over time, making breathing difficult. The main symptoms of COPD include shortness of breath, cough and sputum production. Emphysema and chronic bronchitis are the two most common conditions that make up COPD. Chronic bronchitis is an inflammation of the lining of the bronchial tubes, which carry air to and from the lungs. Emphysema occurs when the air sacs (alveoli) at the end of the smallest air passages (bronchioles) in the lungs are gradually destroyed. COPD causes serious long‐term disability and early death. It is the third leading cause of death in the U.S., affecting more than 12 million Americans.
The Company’s lead development compound, ASM‐024, with its novel mechanism of action, has demonstrated the capabilities of inhibitory effects on inflammation, bronchoprotection and smooth muscle relaxation in pre‐clinical asthma models. ASM‐024 delivered as a solution for nebulization achieved proof of concept in the treatment of patients with asthma in a phase 2 clinical trial program. The company
is now focused on bridging from the ASM‐024 solution formulation to the development of the compound in a dry powder for inhalation (DPI) dosage form.
ASM‐024 demonstrated a highly‐significant effect on FEV₁ and methacholine PC₂₀ pre‐allergen challenge in a phase 2 study utilizing the solution for nebulization formulation. In pre‐clinical asthma models, ASM‐024 has demonstrated an adjunctive benefit when combined with long‐ and short‐acting ß‐agonists and long‐ and short‐acting muscarinic antagonists.
Based on recent findings about the novel mechanism of action for ASM‐024 with nicotinic and muscarinic effects, Asmacure initiated a phase 2 study in COPD patients.